KR20010079526A - 용해성 생분해 유전자 전달 담체인 폴리-l-리신의폴리에스테르 유사체 - Google Patents

용해성 생분해 유전자 전달 담체인 폴리-l-리신의폴리에스테르 유사체 Download PDF

Info

Publication number
KR20010079526A
KR20010079526A KR1020017000489A KR20017000489A KR20010079526A KR 20010079526 A KR20010079526 A KR 20010079526A KR 1020017000489 A KR1020017000489 A KR 1020017000489A KR 20017000489 A KR20017000489 A KR 20017000489A KR 20010079526 A KR20010079526 A KR 20010079526A
Authority
KR
South Korea
Prior art keywords
paga
composition
poly
nucleic acid
dna
Prior art date
Application number
KR1020017000489A
Other languages
English (en)
Korean (ko)
Inventor
종상 박
영훈 최
성완 김
Original Assignee
추후제출
익스프레션 제네틱스, 인코퍼레이티드
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 추후제출, 익스프레션 제네틱스, 인코퍼레이티드 filed Critical 추후제출
Publication of KR20010079526A publication Critical patent/KR20010079526A/ko

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G63/00Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
    • C08G63/02Polyesters derived from hydroxycarboxylic acids or from polycarboxylic acids and polyhydroxy compounds
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1629Organic macromolecular compounds
    • A61K9/1641Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poloxamers
    • A61K9/1647Polyesters, e.g. poly(lactide-co-glycolide)
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B82NANOTECHNOLOGY
    • B82BNANOSTRUCTURES FORMED BY MANIPULATION OF INDIVIDUAL ATOMS, MOLECULES, OR LIMITED COLLECTIONS OF ATOMS OR MOLECULES AS DISCRETE UNITS; MANUFACTURE OR TREATMENT THEREOF
    • B82B1/00Nanostructures formed by manipulation of individual atoms or molecules, or limited collections of atoms or molecules as discrete units
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G63/00Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
    • C08G63/66Polyesters containing oxygen in the form of ether groups
    • C08G63/664Polyesters containing oxygen in the form of ether groups derived from hydroxy carboxylic acids
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G63/00Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
    • C08G63/68Polyesters containing atoms other than carbon, hydrogen and oxygen
    • C08G63/685Polyesters containing atoms other than carbon, hydrogen and oxygen containing nitrogen
    • C08G63/6852Polyesters containing atoms other than carbon, hydrogen and oxygen containing nitrogen derived from hydroxy carboxylic acids
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B82NANOTECHNOLOGY
    • B82YSPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Polymers & Plastics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Polyesters Or Polycarbonates (AREA)
  • Biological Depolymerization Polymers (AREA)
  • Polyamides (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020017000489A 1998-07-13 1999-07-13 용해성 생분해 유전자 전달 담체인 폴리-l-리신의폴리에스테르 유사체 KR20010079526A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US9268298P 1998-07-13 1998-07-13
US60/092,682 1998-07-13
PCT/US1999/015887 WO2000002950A1 (en) 1998-07-13 1999-07-13 Polyester analogue of poly-l-lysine as a soluble, biodegradable gene delivery carrier

Publications (1)

Publication Number Publication Date
KR20010079526A true KR20010079526A (ko) 2001-08-22

Family

ID=22234532

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020017000489A KR20010079526A (ko) 1998-07-13 1999-07-13 용해성 생분해 유전자 전달 담체인 폴리-l-리신의폴리에스테르 유사체

Country Status (15)

Country Link
US (1) US6217912B1 (fi)
EP (1) EP1117720A4 (fi)
JP (1) JP2002520435A (fi)
KR (1) KR20010079526A (fi)
CN (1) CN1313873A (fi)
AU (1) AU5100899A (fi)
BR (1) BR9912070A (fi)
CA (1) CA2337680A1 (fi)
FI (1) FI20010069A (fi)
HK (1) HK1041012A1 (fi)
IL (1) IL140785A0 (fi)
MX (1) MXPA01000271A (fi)
NO (1) NO20010201L (fi)
TR (1) TR200100254T2 (fi)
WO (1) WO2000002950A1 (fi)

Families Citing this family (121)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5610659B2 (ja) * 2000-07-21 2014-10-22 ルバンス セラピュティックス インク.Revance Therapeutics,Inc. 多成分生物学的輸送システム
US20040220100A1 (en) * 2000-07-21 2004-11-04 Essentia Biosystems, Inc. Multi-component biological transport systems
GB0103110D0 (en) * 2000-08-25 2001-03-28 Aventis Pharma Inc A membrane penetrating peptide encoded by a nuclear localization sequence from human period 1
US20040126900A1 (en) * 2001-04-13 2004-07-01 Barry Stephen E High affinity peptide- containing nanoparticles
US7094810B2 (en) * 2001-06-08 2006-08-22 Labopharm, Inc. pH-sensitive block copolymers for pharmaceutical compositions
US6939564B2 (en) * 2001-06-08 2005-09-06 Labopharm, Inc. Water-soluble stabilized self-assembled polyelectrolytes
US7030127B2 (en) 2001-06-29 2006-04-18 Ethicon, Inc. Composition and medical devices utilizing bioabsorbable polymeric waxes
US6967234B2 (en) 2002-12-18 2005-11-22 Ethicon, Inc. Alkyd-lactone copolymers for medical applications
US7034037B2 (en) 2001-06-29 2006-04-25 Ethicon, Inc. Compositions and medical devices utilizing bioabsorbable polymeric waxes and rapamycin
CN1194704C (zh) * 2001-11-12 2005-03-30 华东理工大学 含有活性药物、主链中具有氨基酸的聚酯及其制备方法
US20030186916A1 (en) * 2002-03-12 2003-10-02 Lei Yu Vector for transfection of eukaryotic cells
US7326426B2 (en) 2002-03-29 2008-02-05 Ethicon, Inc. Compositions and medical devices utilizing bioabsorbable liquid polymers
US7005136B2 (en) 2002-03-29 2006-02-28 Ethicon, Inc. Bone replacement materials utilizing bioabsorbable liquid polymers
US7374930B2 (en) * 2002-05-21 2008-05-20 Expression Genetics, Inc. GLP-1 gene delivery for the treatment of type 2 diabetes
US7368125B2 (en) 2002-06-05 2008-05-06 Ethicon, Inc. Amphiphilic polymers for medical applications
US7026374B2 (en) 2002-06-25 2006-04-11 Aruna Nathan Injectable microdispersions for medical applications
US7101566B2 (en) 2002-06-28 2006-09-05 Ethicon, Inc. Polymer coated microparticles for sustained release
WO2004050683A2 (en) * 2002-12-02 2004-06-17 Abgenix, Inc. Antibodies directed to tumor necrosis factor and uses thereof
US6872799B2 (en) 2002-12-18 2005-03-29 Ethicon, Inc. Functionalized polymers for medical applications
US6866860B2 (en) 2002-12-19 2005-03-15 Ethicon, Inc. Cationic alkyd polyesters for medical applications
JP2006523130A (ja) * 2003-03-06 2006-10-12 ジョン エイチ. シャダック, 適合性光学レンズおよび製造方法
US7309757B2 (en) * 2003-06-20 2007-12-18 Agency For Science, Technology And Research Polymers for the delivery of bioactive agents and methods of their preparation
US7883720B2 (en) * 2003-07-09 2011-02-08 Wisconsin Alumni Research Foundation Charge-dynamic polymers and delivery of anionic compounds
CA2558676C (en) * 2004-03-03 2019-04-16 Essentia Biosystems, Inc. Compositions and methods for topical diagnostic and therapeutic transport
NZ592041A (en) 2004-03-03 2012-12-21 Revance Therapeutics Inc Compositions and methods for topical application and transdermal delivery of botulinum toxins
US9211248B2 (en) 2004-03-03 2015-12-15 Revance Therapeutics, Inc. Compositions and methods for topical application and transdermal delivery of botulinum toxins
US20120269886A1 (en) 2004-12-22 2012-10-25 Nitto Denko Corporation Therapeutic agent for pulmonary fibrosis
HUE056941T2 (hu) 2004-12-22 2022-04-28 Nitto Denko Corp Gyógyszerhordozó és gyógyszerhordozó kit fibrózis gátlására
ZA200707352B (en) * 2005-03-03 2009-04-29 Revance Therapeutics Inc Compositions and methods for topical application and transdermal delivery of botulinum toxins
CA2606270A1 (en) * 2005-04-19 2006-10-26 Massachusetts Institute Of Technology Amphiphilic polymers and methods of use thereof
US20070031512A1 (en) * 2005-08-03 2007-02-08 Amcol International Corporation Virus-interacting layered phyllosilicates and methods of inactivating viruses
US20100272769A1 (en) * 2005-08-03 2010-10-28 Amcol International Virus-, Bacteria-, and Fungi-Interacting Layered Phyllosilicates and Methods of Use
US20080184618A1 (en) * 2005-08-03 2008-08-07 Amcol International Virus-Interacting Layered Phyllosilicates and Methods of Use
AU2006315117A1 (en) * 2005-11-17 2007-05-24 Revance Therapeutics, Inc. Compositions and methods of topical application and transdermal delivery of botulinum toxins with reduced non-toxin proteins
US9572886B2 (en) 2005-12-22 2017-02-21 Nitto Denko Corporation Agent for treating myelofibrosis
FR2899231B1 (fr) * 2006-04-03 2008-09-05 Isochem Sa NOUVEAUX O-CARBOXY ANHYDRIDES (OCAs) A FONCTION SALIFIABLE ET POLYMERES OBTENUS A PARTIR DE CES OCAs
US8124598B2 (en) 2006-09-14 2012-02-28 Sharon Sageman 7-keto DHEA for psychiatric use
BRPI0716890A2 (pt) * 2006-09-22 2013-10-22 Labopharm Inc Composição, e, método de produção de uma composição, de administração de um agente farmaceuticamente ativo insolúvel em água a um mamífero, e de tratamento de câncer em um mamífero
MX2009007068A (es) * 2006-12-29 2009-07-10 Revance Therapeutics Inc Composiciones y metodos de aplicacion topica y suministro transdermico de toxinas botulinicas estabilizadas con fragmentos de polipeptido provenientes de hiv-tat.
EP2109363A4 (en) * 2006-12-29 2014-07-09 Revance Therapeutics Inc TRANSPORT MOLECULES USING HIV TAT POLYPEPTIDES WITH REVERSE SEQUENCE
US8834918B2 (en) * 2007-01-22 2014-09-16 Wisconsin Alumni Research Foundation Modified multilayered film
TWI407971B (zh) 2007-03-30 2013-09-11 Nitto Denko Corp Cancer cells and tumor-related fibroblasts
EP2167069B1 (en) * 2007-05-23 2011-10-26 Amcol International Corporation Cholesterol-interacting layered phyllosilicates and methods of reducing hypercholesteremia in a mammal
US20100160274A1 (en) * 2007-09-07 2010-06-24 Sharon Sageman 7-KETO DHEA for Psychiatric Use
JP2010539245A (ja) 2007-09-14 2010-12-16 日東電工株式会社 薬物担体
WO2009049100A2 (en) 2007-10-09 2009-04-16 Wisconsin Alumni Research Foundation Ultrathin multilayered films for controlled release of anionic reagents
US8324333B2 (en) * 2008-06-05 2012-12-04 Wisconsin Alumni Research Foundation Anionic charge-dynamic polymers for release of cationic agents
WO2010093452A2 (en) * 2009-02-11 2010-08-19 University Of Houston Reducible polymers for non-viral gene delivery
US8470891B2 (en) * 2009-12-23 2013-06-25 International Business Machines Corporation Biodegradable block polymers for drug delivery, and methods related thereto
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
HRP20220796T1 (hr) 2010-10-01 2022-10-14 ModernaTX, Inc. Ribonukleinske kiseline koje sadrže n1-metil-pseudouracil i njihove uporabe
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP3492109B1 (en) 2011-10-03 2020-03-04 ModernaTX, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
RS63244B1 (sr) 2011-12-16 2022-06-30 Modernatx Inc Kompozicije modifikovane mrna
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
EP3434774A1 (en) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
GB201304245D0 (en) * 2013-03-08 2013-04-24 Inst Quimic De Sarria Chemical compounds
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP3019619B1 (en) 2013-07-11 2021-08-25 ModernaTX, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
SG11201602503TA (en) 2013-10-03 2016-04-28 Moderna Therapeutics Inc Polynucleotides encoding low density lipoprotein receptor
JP2017500865A (ja) 2013-12-19 2017-01-12 ノバルティス アーゲー レプチンmRNAの組成物および製剤
EP3169693B1 (en) 2014-07-16 2022-03-09 ModernaTX, Inc. Chimeric polynucleotides
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
JP2017522895A (ja) 2014-07-31 2017-08-17 ザ ボード オブ リージェンツ オブ ザ ユニヴァーシティ オブ オクラホマ 哺乳類rpe65の高イソメロヒドロラーゼ活性突然変異体
CN105920618A (zh) * 2016-06-13 2016-09-07 上海交通大学医学院附属新华医院 一种生物靶向纳米基因材料及其制造方法
US11767520B2 (en) 2017-04-20 2023-09-26 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
ES2952779T3 (es) 2017-05-18 2023-11-06 Modernatx Inc ARN mensajero modificado que comprende elementos de ARN funcionales
JP7285220B2 (ja) 2017-05-18 2023-06-01 モデルナティエックス インコーポレイテッド 連結したインターロイキン-12(il12)ポリペプチドをコードするポリヌクレオチドを含む脂質ナノ粒子
US20200268666A1 (en) 2017-06-14 2020-08-27 Modernatx, Inc. Polynucleotides encoding coagulation factor viii
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
JP7423521B2 (ja) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド
JP7424976B2 (ja) 2017-11-22 2024-01-30 モダーナティエックス・インコーポレイテッド プロピオン酸血症の治療用のプロピオニルCoAカルボキシラーゼアルファ及びベータサブユニットをコードするポリヌクレオチド
MA50803A (fr) 2017-11-22 2020-09-30 Modernatx Inc Polynucléotides codant pour l'ornithine transcarbamylase pour le traitement de troubles du cycle de l'urée
MA51523A (fr) 2018-01-05 2020-11-11 Modernatx Inc Polynucléotides codant pour des anticorps anti-virus du chikungunya
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
SG11202012043RA (en) 2018-07-03 2021-01-28 Gilead Sciences Inc Antibodies that target hiv gp120 and methods of use
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
EP3833762A4 (en) 2018-08-09 2022-09-28 Verseau Therapeutics, Inc. OLIGONUCLEOTIDE COMPOSITIONS FOR TARGETING CCR2 AND CSF1R AND THEIR USES
WO2020047201A1 (en) 2018-09-02 2020-03-05 Modernatx, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
EP3849594A2 (en) 2018-09-13 2021-07-21 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
EP3849595A2 (en) 2018-09-13 2021-07-21 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease
MA53615A (fr) 2018-09-14 2021-07-21 Modernatx Inc Polynucléotides codant pour le polypeptide a1, de la famille de l'uridine diphosphate glycosyltransférase 1, pour le traitement du syndrome de crigler-najjar
WO2020069169A1 (en) 2018-09-27 2020-04-02 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220226438A1 (en) 2019-05-08 2022-07-21 Astrazeneca Ab Compositions for skin and wounds and methods of use thereof
CA3142513A1 (en) 2019-06-25 2020-12-30 Gilead Sciences, Inc. Flt3l-fc fusion proteins and methods of use
KR20220047277A (ko) 2019-07-16 2022-04-15 길리애드 사이언시즈, 인코포레이티드 Hiv 백신, 및 이의 제조 및 사용 방법
MX2022003658A (es) 2019-09-30 2022-04-25 Gilead Sciences Inc Vacunas contra el virus de la hepatitis b (vhb) y metodos para tratar el vhb.
EP4158005A1 (en) 2020-06-01 2023-04-05 ModernaTX, Inc. Phenylalanine hydroxylase variants and uses thereof
EP3954393A1 (en) 2020-08-13 2022-02-16 Bioasis Technologies Inc. Combination therapies for delivery across the blood brain barrier
TW202406932A (zh) 2020-10-22 2024-02-16 美商基利科學股份有限公司 介白素2-Fc融合蛋白及使用方法
US20230406895A1 (en) 2020-11-13 2023-12-21 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
JP2024512026A (ja) 2021-03-24 2024-03-18 モデルナティエックス インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療を目的とした脂質ナノ粒子及びオルニチントランスカルバミラーゼをコードするポリヌクレオチド
US20240207374A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
US20240207444A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
US20240175033A1 (en) 2021-03-26 2024-05-30 Mina Therapeutics Limited TMEM173 saRNA Compositions and Methods of Use
EP4355882A2 (en) 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
EP4408871A1 (en) 2021-10-01 2024-08-07 ModernaTX, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
CN114452266B (zh) * 2022-02-09 2023-05-19 南京凯玛生物科技有限公司 一种基于重组核糖体蛋白的核酸药物递送系统及其制备方法和应用
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2024015741A1 (en) 2022-07-12 2024-01-18 Gilead Sciences, Inc. Hiv immunogenic polypeptides and vaccines and uses thereof
WO2024026254A1 (en) 2022-07-26 2024-02-01 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2024197033A1 (en) 2023-03-21 2024-09-26 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of heart failure

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5942253A (en) * 1995-10-12 1999-08-24 Immunex Corporation Prolonged release of GM-CSF
ZA9811377B (en) * 1997-12-12 1999-08-27 Expression Genetics Inc Positively charged poly[alpha-(omega-aminoalkyl) glycolic acid[ for the delivery of a bioactive agent via tissue and cellular uptake.

Also Published As

Publication number Publication date
TR200100254T2 (tr) 2001-06-21
HK1041012A1 (zh) 2002-06-28
WO2000002950A1 (en) 2000-01-20
FI20010069A (fi) 2001-03-09
US6217912B1 (en) 2001-04-17
AU5100899A (en) 2000-02-01
CN1313873A (zh) 2001-09-19
EP1117720A4 (en) 2001-11-14
WO2000002950A9 (en) 2000-07-20
EP1117720A1 (en) 2001-07-25
IL140785A0 (en) 2002-02-10
MXPA01000271A (es) 2002-10-17
BR9912070A (pt) 2001-04-10
NO20010201D0 (no) 2001-01-12
JP2002520435A (ja) 2002-07-09
CA2337680A1 (en) 2000-01-20
NO20010201L (no) 2001-03-12

Similar Documents

Publication Publication Date Title
KR20010079526A (ko) 용해성 생분해 유전자 전달 담체인 폴리-l-리신의폴리에스테르 유사체
ZA200100286B (en) Actuator with approach pre-stroke and working stroke for operating a tool.
US6652886B2 (en) Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
Kim et al. Receptor-mediated gene delivery into antigen presenting cells using mannosylated chitosan/DNA nanoparticles
Garnett Gene-delivery systems using cationic polymers
WO2003063827A1 (en) Biodegradable multi-block copolymers of poly(amino acid)s and poly(ethylene glycol)
JP2004522809A (ja) 生物学的適合遺伝子送達剤としての新規カチオンリポポリマー
EP1448586A2 (en) Therapeutic methods for nucleic acid delivery vehicles
Anwer et al. Recent progress in polymeric gene delivery systems
EP0832269A1 (en) Nucleic acid transporters for delivery of nucleic acids into a cell
KR19990030290A (ko) 생물학적으로 허용되는 저분자량의 폴리에틸렌이민
Karim et al. Scope and challenges of nanoparticle-based mRNA delivery in cancer treatment
CN112107680A (zh) 一种mRNA-脂质体复合物及其应用
US20050118718A1 (en) Stabilization and controlled delivery of ionic biopharmaceuticals
CN114099699A (zh) 一种纳米递送系统及其制备方法和应用
US20060014695A1 (en) Hpma-polyamine conjugates and uses therefore
JP2001526181A (ja) 遺伝子輸送体であるグラフト共重合体
CN114452266B (zh) 一种基于重组核糖体蛋白的核酸药物递送系统及其制备方法和应用
JP4988114B2 (ja) 生物学的材料の送達系
AU725723B2 (en) Composition containing chitosan
KR20010069179A (ko) 유전자 전달용 양이온성 리피드 및 그 제조방법
CN112316153B (zh) 一种抗肿瘤的药物组合物及其应用
JP2003531181A (ja) 核酸を細胞に投与するための粒状複合体
CN118161630A (zh) 药物组合物及其应用
Peluffo Modular multifunctional protein vectors for gene therapy

Legal Events

Date Code Title Description
WITN Application deemed withdrawn, e.g. because no request for examination was filed or no examination fee was paid